CN104684585A - 用于治疗溶酶体贮积症的环糊精 - Google Patents

用于治疗溶酶体贮积症的环糊精 Download PDF

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Publication number
CN104684585A
CN104684585A CN201380052023.0A CN201380052023A CN104684585A CN 104684585 A CN104684585 A CN 104684585A CN 201380052023 A CN201380052023 A CN 201380052023A CN 104684585 A CN104684585 A CN 104684585A
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CN
China
Prior art keywords
cyclodextrin
disease
subject
compound
cholesterol
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Pending
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CN201380052023.0A
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English (en)
Chinese (zh)
Inventor
J·麦丘
W·郑
M·许
M·斯瓦鲁普
J·J·马鲁甘
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US Department of Health and Human Services
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US Department of Health and Human Services
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Priority to CN201711057517.4A priority Critical patent/CN107865968B/zh
Publication of CN104684585A publication Critical patent/CN104684585A/zh
Pending legal-status Critical Current

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/715Polysaccharides, i.e. having more than five saccharide radicals attached to each other by glycosidic linkages; Derivatives thereof, e.g. ethers, esters
    • A61K31/716Glucans
    • A61K31/724Cyclodextrins
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/335Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
    • A61K31/35Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having six-membered rings with one oxygen as the only ring hetero atom
    • A61K31/352Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having six-membered rings with one oxygen as the only ring hetero atom condensed with carbocyclic rings, e.g. methantheline 
    • A61K31/3533,4-Dihydrobenzopyrans, e.g. chroman, catechin
    • A61K31/355Tocopherols, e.g. vitamin E
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/08Drugs for disorders of the metabolism for glucose homeostasis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C08ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
    • C08BPOLYSACCHARIDES; DERIVATIVES THEREOF
    • C08B37/00Preparation of polysaccharides not provided for in groups C08B1/00 - C08B35/00; Derivatives thereof
    • C08B37/0006Homoglycans, i.e. polysaccharides having a main chain consisting of one single sugar, e.g. colominic acid
    • C08B37/0009Homoglycans, i.e. polysaccharides having a main chain consisting of one single sugar, e.g. colominic acid alpha-D-Glucans, e.g. polydextrose, alternan, glycogen; (alpha-1,4)(alpha-1,6)-D-Glucans; (alpha-1,3)(alpha-1,4)-D-Glucans, e.g. isolichenan or nigeran; (alpha-1,4)-D-Glucans; (alpha-1,3)-D-Glucans, e.g. pseudonigeran; Derivatives thereof
    • C08B37/0012Cyclodextrin [CD], e.g. cycle with 6 units (alpha), with 7 units (beta) and with 8 units (gamma), large-ring cyclodextrin or cycloamylose with 9 units or more; Derivatives thereof
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/92Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving lipids, e.g. cholesterol, lipoproteins, or their receptors

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Organic Chemistry (AREA)
  • Hematology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Diabetes (AREA)
  • Biochemistry (AREA)
  • Urology & Nephrology (AREA)
  • Immunology (AREA)
  • Endocrinology (AREA)
  • Obesity (AREA)
  • Biotechnology (AREA)
  • Food Science & Technology (AREA)
  • Polymers & Plastics (AREA)
  • Biophysics (AREA)
  • Pathology (AREA)
  • Cell Biology (AREA)
  • General Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Analytical Chemistry (AREA)
  • Materials Engineering (AREA)
  • Physics & Mathematics (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Emergency Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CN201380052023.0A 2012-08-03 2013-08-03 用于治疗溶酶体贮积症的环糊精 Pending CN104684585A (zh)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CN201711057517.4A CN107865968B (zh) 2012-08-03 2013-08-03 用于治疗溶酶体贮积症的环糊精

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261679668P 2012-08-03 2012-08-03
US61/679,668 2012-08-03
PCT/US2013/053527 WO2014022841A1 (en) 2012-08-03 2013-08-03 Cyclodextrin for the treatment of lysosomal storage diseases

Related Child Applications (1)

Application Number Title Priority Date Filing Date
CN201711057517.4A Division CN107865968B (zh) 2012-08-03 2013-08-03 用于治疗溶酶体贮积症的环糊精

Publications (1)

Publication Number Publication Date
CN104684585A true CN104684585A (zh) 2015-06-03

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CN201380052023.0A Pending CN104684585A (zh) 2012-08-03 2013-08-03 用于治疗溶酶体贮积症的环糊精
CN201711057517.4A Active CN107865968B (zh) 2012-08-03 2013-08-03 用于治疗溶酶体贮积症的环糊精

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US (2) US20150216895A1 (enExample)
EP (1) EP2879714B1 (enExample)
JP (3) JP2015524444A (enExample)
CN (2) CN104684585A (enExample)
AU (2) AU2013296170A1 (enExample)
CA (1) CA2880880C (enExample)
WO (1) WO2014022841A1 (enExample)

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN107847517A (zh) * 2015-06-10 2018-03-27 维瑟公司 羟丙基β‑环糊精组合物及方法
CN109153735A (zh) * 2016-01-21 2019-01-04 阿托恩波罗斯生命科学 基于环糊精的聚合物、其方法、组合物和应用
CN111655291A (zh) * 2017-08-28 2020-09-11 阿斯德拉有限责任公司 环糊精在涉及磷脂失调的疾病和病症中的用途

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2015524444A (ja) 2012-08-03 2015-08-24 ザ ユナイテッド ステイツ オブ アメリカ, アズ リプレゼンテッド バイ ザ セクレタリー, デパートメント オブ ヘルス アンド ヒューマン サービシーズ ライソゾーム蓄積症を治療するためのシクロデキストリン
EP3078379B1 (en) 2013-12-05 2018-11-28 National University Corporation Kumamoto University Drug for the treatment of cholesterol accumulation disorders, and screening method for same
US10413517B2 (en) 2014-06-12 2019-09-17 University Of Notre Dame Du Lac Composition and method for the treatment of neurological diseases and cerebral injury
CN107949380B (zh) * 2015-02-11 2021-07-06 西奈山伊坎医学院 用于尼曼-匹克病和其他溶酶体贮积症的npc1的苯磺酰胺上调剂
TWI815796B (zh) 2016-08-02 2023-09-21 美商杜瑞克公司 包含5-膽甾烯-3,25-二醇3-硫酸酯(25hc3s)或其醫藥上可接受之鹽及至少一種環狀寡醣的組成物,以及使用彼等之方法
JP2019533011A (ja) * 2016-09-19 2019-11-14 アトン ポロス ライフサイエンシーズ シクロデキストリン系ポリマー、方法、組成物及びその応用
JPWO2018199287A1 (ja) * 2017-04-28 2020-03-12 国立大学法人京都大学 クリスタリン網膜症の処置および/または予防方法
CA3076821A1 (en) 2017-09-28 2019-04-04 Asdera Llc Use of cyclodextrins in diseases and disorders involving phospholipid dysregulation
ES2992137T3 (es) 2018-10-29 2024-12-09 Cyclo Therapeutics Inc Métodos para tratar la enfermedad de Alzheimer con HP-[ß]-ciclodextrina
CN113490691A (zh) 2019-01-03 2021-10-08 劣势者药物有限公司 环糊精二聚体、其组合物及其用途
JP7264452B2 (ja) * 2019-04-15 2023-04-25 塩水港精糖株式会社 ライソゾーム病の治療または予防のための医薬組成物
US12226431B2 (en) 2021-09-11 2025-02-18 Porosome Therapeutics, Inc. Aerosol and topical administration of a formulation containing cyclodextrin, quercetin and zinc, in combination or separately, to mitigate infection by enveloped viruses
JP7551340B2 (ja) * 2020-06-04 2024-09-17 株式会社三共 遊技機
JP7551339B2 (ja) * 2020-06-04 2024-09-17 株式会社三共 遊技機
WO2022051631A1 (en) * 2020-09-03 2022-03-10 University Of Notre Dame Du Lac Therapy to stimulate hippocampal neural progenitors and adult neurogenesis
US11446325B2 (en) * 2020-09-30 2022-09-20 Cyclolab Cyclodextrin Research And Development Laboratory Ltd. Cyclodextrin derivatives in the treatment or prevention of lysosomal neurodegenerative diseases
IL315052A (en) 2022-02-18 2024-10-01 Beren Therapeutics P B C Compositions of hydroxypropyl-beta-cyclodextrin and methods of purifying the same
WO2025090588A1 (en) 2023-10-24 2025-05-01 Minerva Neurosciences, Inc. Roluperidone for the treatment of lysosomal storage disorders and their symptoms

Family Cites Families (16)

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US4727064A (en) 1984-04-25 1988-02-23 The United States Of America As Represented By The Department Of Health And Human Services Pharmaceutical preparations containing cyclodextrin derivatives
US6407079B1 (en) 1985-07-03 2002-06-18 Janssen Pharmaceutica N.V. Pharmaceutical compositions containing drugs which are instable or sparingly soluble in water and methods for their preparation
US5376645A (en) 1990-01-23 1994-12-27 University Of Kansas Derivatives of cyclodextrins exhibiting enhanced aqueous solubility and the use thereof
US5262404A (en) 1990-02-15 1993-11-16 The Trustees Of The University Of Pennsylvania/Childrens Hospital Corporation Cyclodextrin polymers and cyclodextrins immobilized on a solid surface
DE69719834T2 (de) 1996-04-24 2003-11-20 Pfizer Inc., New York Dentalwerkstoffe enthaltend Cyclodextrine und phenolische Verbindungen
FR2767834B1 (fr) 1997-08-29 1999-12-03 Inst Francais Du Petrole Mono et di-derives de cyclodextrines, leurs synthese et purification et leur utilisation en support
AU2001259725A1 (en) 2000-05-15 2001-11-26 The Procter And Gamble Company Compositions comprising cyclodextrin derivatives
US7442372B2 (en) * 2003-08-29 2008-10-28 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
WO2005112637A1 (en) * 2004-05-14 2005-12-01 Decode Chemistry, Inc. Formulations for non-parenteral use including hydrophobic cyclodextrins
EP1655034A1 (en) 2004-10-10 2006-05-10 Université de Liège Use of cyclodextrin for treatment and prevention of bronchial inflammatory diseases.
NZ573132A (en) * 2006-06-06 2012-05-25 Glaxo Group Ltd Administration of anti-cd3 antibodies in the treatment of autoimmune diseases
WO2010138802A2 (en) * 2009-05-28 2010-12-02 Cornell University Compositions and their use for removing cholesterol
WO2012012473A1 (en) * 2010-07-19 2012-01-26 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Use of delta tocopherol for the treatment of lysosomal storage disorders
US10463687B2 (en) * 2011-01-20 2019-11-05 Cornell University Treatments for retinal disorders
JP2015524444A (ja) 2012-08-03 2015-08-24 ザ ユナイテッド ステイツ オブ アメリカ, アズ リプレゼンテッド バイ ザ セクレタリー, デパートメント オブ ヘルス アンド ヒューマン サービシーズ ライソゾーム蓄積症を治療するためのシクロデキストリン
FR3014694B1 (fr) 2013-12-13 2016-11-11 Roquette Freres Compositions a base de methyl-cyclodextrines pour le traitement et/ou la prevention de maladies par augmentation du taux de cholesterol-hdl

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN107847517A (zh) * 2015-06-10 2018-03-27 维瑟公司 羟丙基β‑环糊精组合物及方法
CN109153735A (zh) * 2016-01-21 2019-01-04 阿托恩波罗斯生命科学 基于环糊精的聚合物、其方法、组合物和应用
CN111655291A (zh) * 2017-08-28 2020-09-11 阿斯德拉有限责任公司 环糊精在涉及磷脂失调的疾病和病症中的用途

Also Published As

Publication number Publication date
AU2013296170A1 (en) 2015-03-12
JP6858286B2 (ja) 2021-04-14
AU2018202964B2 (en) 2019-08-22
US20150216895A1 (en) 2015-08-06
CN107865968A (zh) 2018-04-03
JP2020097620A (ja) 2020-06-25
JP2015524444A (ja) 2015-08-24
JP2018162277A (ja) 2018-10-18
CA2880880A1 (en) 2014-02-06
US11020422B2 (en) 2021-06-01
WO2014022841A1 (en) 2014-02-06
EP2879714B1 (en) 2021-04-14
US20180110798A1 (en) 2018-04-26
EP2879714A1 (en) 2015-06-10
HK1210960A1 (en) 2016-05-13
CN107865968B (zh) 2021-03-05
CA2880880C (en) 2021-01-26
AU2018202964A1 (en) 2018-05-17

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